PackGene and GC4K Team Up Along with Weill Cornell Medicine for Gene Therapy to Treat Rare Neurological Disease

Nora was 4 years old when she was diagnosed. She has Pkan's disease, a rare neurological degeneration, which progressively attacks the muscles and then the vision. A disease for which there is no treatment. Nora is now in a wheelchair, but she has never let go of her smile. It's his armour against adversity. To help him, an online fundraiser is open.

PackGene and GC4K Team Up Along with Weill Cornell Medicine for Gene Therapy to Treat Rare Neurological Disease

Through this global alliance, PackGene, Weill Cornell Medicine, and GC4K, an Australian non-profit, intend to deliver a custom-tailored gene therapy solution to treat hereditary spastic paraplegia type 56, a particularly rare neurological disease.